“Facioscapulohumeral Muscular Dystrophy Pipeline Insight, 2025“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Facioscapulohumeral Muscular Dystrophy Market.
The Facioscapulohumeral Muscular Dystrophy Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.
Some of the key takeaways from the Facioscapulohumeral Muscular Dystrophy Pipeline Report:
Companies across the globe are diligently working toward developing novel Facioscapulohumeral Muscular Dystrophy treatment therapies with a considerable amount of success over the years.
Facioscapulohumeral Muscular Dystrophy companies working in the treatment market are Dyne Therapeutics, miRecule, Avidity Biosciences, aTyr Pharma, Fulcrum Therapeutics, and others, are developing therapies for the Facioscapulohumeral Muscular Dystrophy treatment
Emerging Facioscapulohumeral Muscular Dystrophy therapies in the different phases of clinical trials are- DYNE-301, MC-DX4, AOC 1020, ATYR 1940, Losmapimod, and others are expected to have a significant impact on the Facioscapulohumeral Muscular Dystrophy market in the coming years.
In April 2025, Epicrispr Biotechnologies, a biotechnology firm focused on developing curative therapies, has received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) for its candidate EPI-321. This innovative treatment represents the first epigenetic-based approach targeting facioscapulohumeral muscular dystrophy (FSHD), a genetic neuromuscular condition.
In March 2025, Epicrispr Biotechnologies has secured $68 million in the initial closing of its Series B funding round to initiate clinical trials for its disease-modifying therapy, EPI-321, targeting facioscapulohumeral muscular dystrophy (FSHD). The funds will also support the clinical advancement of this treatment for the genetic neuromuscular disorder, along with the continued development of the company’s broader therapeutic pipeline.
In March 2025, Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company advancing a new class of RNA-based medicines known as Antibody Oligonucleotide Conjugates (AOCs™), has announced the completion of enrollment for the biomarker cohort in its Phase 1/2 FORTITUDE™ clinical trial. The study is evaluating delpacibart braxlosiran (del-brax) in individuals with facioscapulohumeral muscular dystrophy (FSHD), with a total of 51 participants enrolled in this cohort.
In October 2024, Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company advancing RNA therapeutics through Antibody Oligonucleotide Conjugates (AOCs™), has launched a biomarker cohort in the Phase 1/2 FORTITUDE™ trial of delpacibart braxlosiran (del-brax/AOC 1020) for individuals with facioscapulohumeral muscular dystrophy (FSHD). The company is exploring a potential accelerated approval pathway for del-brax, with plans to complete biomarker cohort enrollment by mid-2025. Additionally, a functional cohort is set to begin in the first half of 2025, and enrollment for the FORTITUDE Open-label Extension (OLE) study is ongoing.
Facioscapulohumeral Muscular Dystrophy Overview
Facioscapulohumeral Muscular Dystrophy (FSHD) is a neuromuscular disorder characterized by progressive muscle weakness and wasting. It primarily affects the muscles of the face (facio), shoulders (scapulo), and upper arms (humeral), although it can also involve muscles in other parts of the body.
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Emerging Facioscapulohumeral Muscular Dystrophy Drugs Under Different Phases of Clinical Development Include:
DYNE-301: Dyne Therapeutics
MC-DX4: miRecule
AOC 1020: Avidity Biosciences
ATYR 1940: aTyr Pharma
Losmapimod: Fulcrum Therapeutics
Facioscapulohumeral Muscular Dystrophy Route of Administration
Facioscapulohumeral Muscular Dystrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as
Oral
Parenteral
Intravenous
Subcutaneous
Topical
Facioscapulohumeral Muscular Dystrophy Molecule Type
Facioscapulohumeral Muscular Dystrophy Products have been categorized under various Molecule types, such as
Monoclonal Antibody
Peptides
Polymer
Small molecule
Gene therapy
Facioscapulohumeral Muscular Dystrophy Pipeline Therapeutics Assessment
Facioscapulohumeral Muscular Dystrophy Assessment by Product Type
Facioscapulohumeral Muscular Dystrophy By Stage and Product Type
Facioscapulohumeral Muscular Dystrophy Assessment by Route of Administration
Facioscapulohumeral Muscular Dystrophy By Stage and Route of Administration
Facioscapulohumeral Muscular Dystrophy Assessment by Molecule Type
Facioscapulohumeral Muscular Dystrophy by Stage and Molecule Type
DelveInsight’s Facioscapulohumeral Muscular Dystrophy Report covers around 10+ products under different phases of clinical development like
Late-stage products (Phase III)
Mid-stage products (Phase II)
Early-stage product (Phase I)
Pre-clinical and Discovery stage candidates
Discontinued & Inactive candidates
Route of Administration
Further Facioscapulohumeral Muscular Dystrophy product details are provided in the report. Download the Facioscapulohumeral Muscular Dystrophy pipeline report to learn more about the emerging Facioscapulohumeral Muscular Dystrophy therapies
Some of the key companies in the Facioscapulohumeral Muscular Dystrophy Therapeutics Market include:
Key companies developing therapies for Facioscapulohumeral Muscular Dystrophy are – Fulcrum Therapeutics, Dyne Therapeutics, GSK, Roche, Facio Therapies, Myocea, Avidity Biosciences, and others.
Facioscapulohumeral Muscular Dystrophy Pipeline Analysis:
The Facioscapulohumeral Muscular Dystrophy pipeline report provides insights into
The report provides detailed insights about companies that are developing therapies for the treatment of Facioscapulohumeral Muscular Dystrophy with aggregate therapies developed by each company for the same.
It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Facioscapulohumeral Muscular Dystrophy Treatment.
Facioscapulohumeral Muscular Dystrophy key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
Facioscapulohumeral Muscular Dystrophy Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Facioscapulohumeral Muscular Dystrophy market.
The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.
Download Sample PDF Report to know more about Facioscapulohumeral Muscular Dystrophy drugs and therapies
Facioscapulohumeral Muscular Dystrophy Pipeline Market Drivers
Advancement in medical technology, investigation into the molecular consequences of DUX4 expression, demand for effective treatment options and novel therapies are some of the important factors that are fueling the Facioscapulohumeral Muscular Dystrophy Market.
Facioscapulohumeral Muscular Dystrophy Pipeline Market Barriers
However, lack of approved therapies, complications involved with facioscapulohumeral muscular dystrophy and other factors are creating obstacles in the Facioscapulohumeral Muscular Dystrophy Market growth.
Scope of Facioscapulohumeral Muscular Dystrophy Pipeline Drug Insight
Coverage: Global
Key Facioscapulohumeral Muscular Dystrophy Companies: Dyne Therapeutics, miRecule, Avidity Biosciences, aTyr Pharma, Fulcrum Therapeutics, and others
Key Facioscapulohumeral Muscular Dystrophy Therapies: DYNE-301, MC-DX4, AOC 1020, ATYR 1940, Losmapimod, and others
Facioscapulohumeral Muscular Dystrophy Therapeutic Assessment: Facioscapulohumeral Muscular Dystrophy current marketed and Facioscapulohumeral Muscular Dystrophy emerging therapies
Facioscapulohumeral Muscular Dystrophy Market Dynamics: Facioscapulohumeral Muscular Dystrophy market drivers and Facioscapulohumeral Muscular Dystrophy market barriers
Request for Sample PDF Report for Facioscapulohumeral Muscular Dystrophy Pipeline Assessment and clinical trials
Table of Contents
1. Facioscapulohumeral Muscular Dystrophy Report Introduction
2. Facioscapulohumeral Muscular Dystrophy Executive Summary
3. Facioscapulohumeral Muscular Dystrophy Overview
4. Facioscapulohumeral Muscular Dystrophy- Analytical Perspective In-depth Commercial Assessment
5. Facioscapulohumeral Muscular Dystrophy Pipeline Therapeutics
6. Facioscapulohumeral Muscular Dystrophy Late Stage Products (Phase II/III)
7. Facioscapulohumeral Muscular Dystrophy Mid Stage Products (Phase II)
8. Facioscapulohumeral Muscular Dystrophy Early Stage Products (Phase I)
9. Facioscapulohumeral Muscular Dystrophy Preclinical Stage Products
10. Facioscapulohumeral Muscular Dystrophy Therapeutics Assessment
11. Facioscapulohumeral Muscular Dystrophy Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Facioscapulohumeral Muscular Dystrophy Key Companies
14. Facioscapulohumeral Muscular Dystrophy Key Products
15. Facioscapulohumeral Muscular Dystrophy Unmet Needs
16 . Facioscapulohumeral Muscular Dystrophy Market Drivers and Barriers
17. Facioscapulohumeral Muscular Dystrophy Future Perspectives and Conclusion
18. Facioscapulohumeral Muscular Dystrophy Analyst Views
19. Appendix
20. About DelveInsight
About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.
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